PE20211197A1 - OLIGONUCLEOTIDES TO MODULATE THE EXPRESSION OF TMEM106B - Google Patents
OLIGONUCLEOTIDES TO MODULATE THE EXPRESSION OF TMEM106BInfo
- Publication number
- PE20211197A1 PE20211197A1 PE2020001186A PE2020001186A PE20211197A1 PE 20211197 A1 PE20211197 A1 PE 20211197A1 PE 2020001186 A PE2020001186 A PE 2020001186A PE 2020001186 A PE2020001186 A PE 2020001186A PE 20211197 A1 PE20211197 A1 PE 20211197A1
- Authority
- PE
- Peru
- Prior art keywords
- oligonucleotide
- nucleotides
- tmem106b
- oligonucleotides
- modulate
- Prior art date
Links
Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
Abstract
SE REFIERE A UN OLIGONUCLEOTIDO ANTISENTIDO MONOCATENARIO DE 10 A 50 NUCLEOTIDOS DE LONGITUD, QUE COMPRENDE UNA SECUENCIA DE NUCLEOTIDOS CONTIGUOS DE 10 A 30 NUCLEOTIDOS DE LONGITUD QUE ES AL MENOS 90 % COMPLEMENTARIO DE UN ACIDO NUCLEICO DIANA DE LA PROTEINA TRANSMEMBRANA 106B DE MAMIFERO (TMEM106B); EN DONDE EL OLIGONUCLEOTIDO ES UN siARN O shARN O FORMA LA CADENA GUIA DE UN COMPLEJO DE siARN O shARN. TAMBIEN SE REFIERE A UNA COMPOSICION FARMACEUTICA QUE COMPRENDE DICHO OLIGONUCLEOTIDO QUE ES UTIL EN EL TRATAMIENTO DE ENFERMEDADES NEURODEGENERATIVAS COMO DEGENERACION LOBULAR FRONTOTEMPORAL (FTLD), ENFERMEDAD DE PARKINSON, LEUCODISTROFIAS HIPOMIELINIZANTES, ENFERMEDAD DE ALZHEIMER.REFERS TO A MONOCATENARY ANTI-SENSE OLIGONUCLEOTIDE OF 10 TO 50 NUCLEOTIDES IN LENGTH, WHICH INCLUDES A SEQUENCE OF CONTIGUOUS NUCLEOTIDES OF 10 TO 30 NUCLEOTIDES IN LENGTH WHICH IS AT LEAST 90% COMPLEMENTARY OF AN ACIMENDUM NUCLEOTIDE 10 TM OF 10 ); WHERE THE OLIGONUCLEOTIDE IS A siARN OR shARN OR IT FORMS THE GUIDE CHAIN OF A siRNA OR shARN COMPLEX. IT ALSO REFERS TO A PHARMACEUTICAL COMPOSITION THAT INCLUDES SAID OLIGONUCLEOTIDE THAT IS USEFUL IN THE TREATMENT OF NEURODEGENERATIVE DISEASES SUCH AS FRONTOTEMPORAL LOBULAR DEGENERATION (FTLD), PARKINSON'S DISEASE, ALLEYPODYCODYSTROPHY, LEIPODYCODISTROPHY, LEIPODYCODISTROPHY, AND PARKINSON'S DISEASE.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP18156142 | 2018-02-09 | ||
US201862669251P | 2018-05-09 | 2018-05-09 | |
PCT/EP2019/053116 WO2019154979A1 (en) | 2018-02-09 | 2019-02-08 | Oligonucleotides for modulating tmem106b expression |
Publications (1)
Publication Number | Publication Date |
---|---|
PE20211197A1 true PE20211197A1 (en) | 2021-07-01 |
Family
ID=65433647
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2020001186A PE20211197A1 (en) | 2018-02-09 | 2019-02-08 | OLIGONUCLEOTIDES TO MODULATE THE EXPRESSION OF TMEM106B |
Country Status (17)
Country | Link |
---|---|
US (1) | US11485975B2 (en) |
EP (1) | EP3749766A1 (en) |
JP (1) | JP7281474B2 (en) |
KR (1) | KR20200108315A (en) |
CN (1) | CN111868245A (en) |
AU (1) | AU2019219194A1 (en) |
BR (1) | BR112020016170A2 (en) |
CA (1) | CA3088071A1 (en) |
CL (1) | CL2020002038A1 (en) |
CR (1) | CR20200346A (en) |
IL (1) | IL276357A (en) |
MA (1) | MA51795A (en) |
MX (1) | MX2020008290A (en) |
PE (1) | PE20211197A1 (en) |
RU (1) | RU2020125769A (en) |
SG (1) | SG11202007093YA (en) |
WO (1) | WO2019154979A1 (en) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP4058152A1 (en) * | 2019-11-14 | 2022-09-21 | The Board of Regents of the University of Oklahoma | Oligonucleotide interference treatments of prostate cancer |
JP2023527684A (en) * | 2020-05-11 | 2023-06-30 | ジェネンテック, インコーポレイテッド | Complement component C1S inhibitors for treating neurological disorders and related compositions, systems and methods of using same |
WO2023141507A1 (en) * | 2022-01-20 | 2023-07-27 | Genentech, Inc. | Antisense oligonucleotides for modulating tmem106b expression |
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-
2019
- 2019-02-08 KR KR1020207022883A patent/KR20200108315A/en not_active Application Discontinuation
- 2019-02-08 CR CR20200346A patent/CR20200346A/en unknown
- 2019-02-08 WO PCT/EP2019/053116 patent/WO2019154979A1/en unknown
- 2019-02-08 AU AU2019219194A patent/AU2019219194A1/en not_active Abandoned
- 2019-02-08 JP JP2020542377A patent/JP7281474B2/en active Active
- 2019-02-08 MA MA051795A patent/MA51795A/en unknown
- 2019-02-08 MX MX2020008290A patent/MX2020008290A/en unknown
- 2019-02-08 BR BR112020016170-5A patent/BR112020016170A2/en not_active Application Discontinuation
- 2019-02-08 CA CA3088071A patent/CA3088071A1/en not_active Abandoned
- 2019-02-08 SG SG11202007093YA patent/SG11202007093YA/en unknown
- 2019-02-08 CN CN201980020478.1A patent/CN111868245A/en active Pending
- 2019-02-08 PE PE2020001186A patent/PE20211197A1/en unknown
- 2019-02-08 RU RU2020125769A patent/RU2020125769A/en not_active Application Discontinuation
- 2019-02-08 EP EP19705306.9A patent/EP3749766A1/en active Pending
-
2020
- 2020-07-28 IL IL276357A patent/IL276357A/en unknown
- 2020-08-05 CL CL2020002038A patent/CL2020002038A1/en unknown
- 2020-08-06 US US16/987,030 patent/US11485975B2/en active Active
Also Published As
Publication number | Publication date |
---|---|
KR20200108315A (en) | 2020-09-17 |
WO2019154979A1 (en) | 2019-08-15 |
CN111868245A (en) | 2020-10-30 |
CR20200346A (en) | 2020-10-19 |
US20210054383A1 (en) | 2021-02-25 |
IL276357A (en) | 2020-09-30 |
CA3088071A1 (en) | 2019-08-15 |
JP2021514182A (en) | 2021-06-10 |
JP7281474B2 (en) | 2023-05-25 |
AU2019219194A1 (en) | 2020-08-06 |
BR112020016170A2 (en) | 2020-12-15 |
US11485975B2 (en) | 2022-11-01 |
RU2020125769A (en) | 2022-03-10 |
RU2020125769A3 (en) | 2022-03-10 |
SG11202007093YA (en) | 2020-08-28 |
MX2020008290A (en) | 2020-09-25 |
EP3749766A1 (en) | 2020-12-16 |
MA51795A (en) | 2020-12-16 |
CL2020002038A1 (en) | 2020-10-23 |
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